BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.
Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in the pre-specified primary analysis in participants from a prior non-interventional study.
Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII infusion rate in the rollover population by 133 infusions per year or 98% from baseline.
Overall, valoctocogene roxaparvovec has continued to be well tolerated.
No participants developed inhibitors to Factor VIII, malignancy, or thromboembolic events.
The European Medicines Agency validated BioMarin's resubmission of a marketing application. A Committee for Medicinal Products for Human Use and Committee for Advanced Therapies opinion is anticipated in 1H of 2022.
In the U.S., BioMarin intends to submit two-year follow-up safety and efficacy data GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the FDA.
The Company plans to meet with FDA to discuss the resubmission of a marketing application targeted for Q2 2022, followed by an expected six-month review by the FDA.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.